The 113th Congress and Medical Research Funding: A Perfect Storm Approaching? | 20 years and $2B later, FDA approves gene-silencing drug | 23andMe expands collaboration with Genentech
January 31, 2013
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Drugmaker partners with patients to develop orphan drug
Staff at NPS Pharmaceuticals in New Jersey worked closely with a 26-year-old Nebraska man to develop a drug to treat his short bowel syndrome. The drugmaker contributed to the patient's Short Bowel Syndrome Foundation, and executives sought his input on marketing the drug, Gattex, to his social network of patients and caregivers. The company's patient care coordinators will help ensure access to the drug, even writing appeal letters if an insurer denies coverage. The New York Times (free-article access for SmartBrief readers) (1/30)
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News from FasterCures
The 113th Congress and Medical Research Funding: A Perfect Storm Approaching?
Wednesday, Feb. 20
1 p.m. to 2 p.m. Eastern
This is no garden-variety budget year in Washington: a tsunami of budget and fiscal issues threaten federal science programs, including the postponed "sequester," the drafting of the fiscal year 2014 budgets and likely fights over the debt ceiling. This FREE webinar will tell you what you need to know about what could happen on Capitol Hill, when, and who the important players are, including some new faces. Register today. Speakers include:
  • Ceci Connolly, managing director, Health Research Institute, PricewaterhouseCoopers.
  • Sudip S. Parikh, Ph.D., vice president and director, Center for Analytics & Public Health, Battelle.
  • Carrie D. Wolinetz, Ph.D., associate vice president for federal relations, Association of American Universities.
  • Moderator: Margaret Anderson, executive director, FasterCures.
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Science and Technology
20 years and $2B later, FDA approves gene-silencing drug
The FDA approved antisense drug Kynamro to treat homozygous familial hypercholesterolemia, a rare, inherited disorder. The technology on which the drug is based was initially developed by Gilead Sciences, which sold its patents to Isis Pharmaceuticals. Sarepta Therapeutics and Prosensa are developing gene-silencing drugs to treat muscular dystrophy. The New York Times (free-article access for SmartBrief readers) (1/29), Xconomy/San Diego (1/29)
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23andMe expands collaboration with Genentech
23andMe is asking for saliva samples from any U.S. patient who used Avastin before this year to treat certain metastatic cancers. Study participants will be asked to send a saliva sample and may also submit a blood sample for genetic analysis, and will complete five online surveys throughout the year. The study, called InVite, aims to identify new genetic markers for the diseases and to understand differences in patient response. GenomeWeb Daily News (free registration) (1/29)
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Amgen, Genzyme to develop new intracellular production methods
Genzyme and Amgen are pushing to develop cheaper and faster ways of manufacturing biological drugs inside cells. Amgen is working on a new process for protein production, while Genzyme is pursuing a concept called continuous manufacturing. MIT Technology Review online (1/30)
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Finance and Economics
L.A. billionaire founds new 'omics company
Dr. Patrick Soon-Shiong, who founded two drug companies, including Abraxis BioScience, has started a new company named NantOmics to change the way cancer is treated. The company aims to create a convergence of genomics technology and social networks to amass and manage data that leads to personalized treatment with kinase inhibitors. Under the NantOmics model, cancer will be treated based not on where it is in the body but on its genetic composition. Bloomberg (1/28), Bloomberg Television (1/28)
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Companion diagnostics change cancer drug development
Qiagen filed its application for FDA approval of a test for epidermal growth factor receptor in lung tumors simultaneously with Boehringer Ingelheim's request for approval of a drug that targets the gene mutation. Diagnostic tests help clinicians determine whether drugs that target specific gene mutations will work in individual patients, but insurers hesitate to reimburse diagnostics in the absence of proof that they improve drug efficacy. When tests are developed and approved in tandem with drugs, reimbursement is more likely, says Qiagen?s Stephen Little. Xconomy/Boston (1/31)
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2012 was good, but not great, for biotech VC
Upfront deal values for venture capital-backed biotech mergers and acquisitions totaled about $3.5 billion last year, which is lower than in 2011 but better than 2008 to 2010, according to data from HBM Partners. Total annual VC-backed private acquisitions have held steady since 2005. The time period from founding to trade sale appears to have risen from about five years in 2005 to nine years in 2012, notes Atlas Venture partner Bruce Booth. Forbes (1/30)
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Orphan drug applications skyrocket as Big Pharma carves out niche
Rare diseases are attracting renewed interest as multinational drugmakers seek to take advantage of easier approval pathways and high prices for orphan drugs. Firms including Sanofi, Pfizer and GlaxoSmithKlein are working on or have already secured approval for drugs that treat rare conditions. The progress in understanding the molecular base for these diseases has also spurred drugmakers' interest. The Wall Street Journal (tiered subscription model) (1/30)
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Policy and Regulation
Researchers find significant overlap in grants
Government agencies may have awarded about $70 million in overlapping funds over the past decade, researchers report in Nature. Critics say the funds could have been used to further other research, but recipients say they used the grants to fund different research. The NIH, National Science Foundation and Department of Defense, among others, have differing standards for applicants who submit grant requests to multiple agencies. Nature/News (1/30)
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Society and Ethics
Scientific communication crisis threatens progress
The science community has long had a reputation for having poor public communication skills, but lately scientists have failed to communicate with one another, writes David Rubenson, Stanford Cancer Institute's associate director for administration and strategic planning. Presentations have become incomprehensible because researchers lack preparation time and large conferences make audience questions difficult to field; the number of scientific publications has exploded; research institutions have expanded; specialization has increased; and funds have declined. "It is up to our scientific leaders at the national institutes, foundations, and academic centers to recognize this problem and realign priorities and goals appropriately," Rubenson writes. The Scientist online (1/30)
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FasterCures is an action tank that works across sectors and diseases to improve the effectiveness and efficiency of the medical research enterprise. FasterCures, a center of the Milken Institute, is nonpartisan and independent of interest groups.
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